Gene Therapy & Genomic Medicine

CRISPR edits, delivery vectors, rare‑disease trials, and approvals.

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Latest in Gene Therapy & Genomic Medicine

BIOENGINEER.ORG
bioengineer.org > aptamer-guided-crispr-cas9-delivery-could-make-cancer-genome-editing-precise

Aptamer-Guided CRISPR-Cas9 Delivery Could Make Cancer Genome Editing

3+ hour, 58+ min ago   (55+ words) Gene editing has long promised a revolution in cancer medicine, yet the promise has remained stubbornly out of reach for one deceptively simple reason: getting the CRISPR-Cas9 machinery into tumor cells, and only tumor cells, is extraordinarily difficult. A new…...

BIOENGINEER.ORG
bioengineer.org > short-rna-reads-underestimate-a-to-i-rna-editing-long-read-sequencing-reveals

Short RNA Reads Underestimate A-to-I RNA Editing, Long-Read Sequencing

9+ hour, 41+ min ago   (89+ words) A widespread blind spot in one of genomics’ most routine measurements has been brought into sharp focus by a new study comparing short-read and long-read transcriptome sequencing. Researchers report that next-generation sequencing, the short-read workhorse of modern RNA analysis, systematically…...

BIOENGINEER.ORG
bioengineer.org > dna-binding-protein-fusions-supercharge-pam-flexible-high-precision-base-editing

DNA-Binding Protein Fusions Supercharge PAM-Flexible, High-Precision Base

10+ hour, 58+ min ago   (77+ words) Scientists have unveiled a significantly upgraded CRISPR base editing platform that combines near-unrestricted DNA targeting with pinpoint accuracy and markedly higher efficiency, potentially transforming efforts to correct disease-causing mutations and engineer improved crops. In a study published in Advanced Science,…...

BIOENGINEER.ORG
bioengineer.org > prime-editing-gets-a-size-upgrade-researchers-insert-large-dna-fragments-with-precision

Prime Editing Gets a Size Upgrade: Researchers Insert Large DNA Fragments

13+ hour, 50+ min ago   (653+ words) The gene-editing field has long faced a stubborn trade-off. Tools such as CRISPR-Cas9 excel at cutting DNA and at making small, targeted changes, but installing large pieces of genetic material into a genome at a precise location — without causing collateral…...

BIOENGINEER.ORG
bioengineer.org > cryo-em-reveals-how-cas12a-uses-a-dna-guide-to-hunt-rna-targets

Cryo-EM Reveals How Cas12a Uses a DNA Guide to Hunt RNA Targets

14+ hour, 54+ min ago   (741+ words) The CRISPR world has long been organized around a simple division of labor: some Cas enzymes are steered by RNA guides to cut DNA, while others are loaded with DNA guides to find RNA....

BIOENGINEER.ORG
bioengineer.org > simpler-crispr-cas9-system-speeds-genome-editing-in-bacillus-bacteria

Simpler CRISPR-Cas9 System Speeds Genome Editing in Bacillus Bacteria

17+ hour, 12+ min ago   (55+ words) Researchers in Germany have unveiled a streamlined genome-editing tool that could make it considerably easier to engineer Bacillus bacteria, a group of microbes that sits at the heart of numerous industrial processes, from enzyme production to the manufacture of vitamins…...

BIOENGINEER.ORG
bioengineer.org > placenta-gene-phlda2-hits-female-fetuses-harder-mouse-study-finds

Placenta Gene PHLDA2 Hits Female Fetuses Harder, Mouse Study Finds

17+ hour, 33+ min ago   (607+ words) Deep inside the mammalian placenta, a quiet genetic tug-of-war shapes how much a growing fetus asks of its mother....

BIOENGINEER.ORG
bioengineer.org > crispr-and-surrogate-broodstock-push-aquaculture-toward-programmable-monosex-and-sterile-fish

CRISPR and Surrogate Broodstock Push Aquaculture Toward Programmable

18+ hour, 29+ min ago   (747+ words) Aquaculture now supplies more than half of the seafood consumed worldwide, yet the industry still struggles with one of biology’s oldest problems: controlling when and how farmed animals reproduce....

BIOENGINEER.ORG
bioengineer.org > crispr-methylation-editing-rewrites-the-cancer-epigenome-toward-causation

CRISPR Methylation Editing Rewrites the Cancer Epigenome Toward Causation

1+ day, 1+ hour ago   (67+ words) Cancer has long been understood as a disease of the genome, driven by mutations that rewire the instructions encoded in DNA. Over the past three decades, however, a parallel truth has crystallized: cancer is equally a disease of the epigenome,…...